Speaker Profile

A parent who took a leadership role in the development of a novel therapy for patients with Angelman Syndrome and her advocacy to transform rare disease treatment in children

DVM, DACVIM, Chief Science Officer, FAST

Dr. Allyson Berent is a veterinary clinical trialist and interventional endoscopist, while serving as the Chief Science Officer at the Foundation for Angelman Syndrome (AS) Therapeutics (FAST) and the Co-Director of the Angelman Syndrome Biomarker and Outcome Measure Consortium (ABOM). She also serves as the Chief Development Officer for Mahzi Therapeutics. Allyson was the co-founder of GeneTx Biotherapeutics, a company started out of FAST to advance a novel antisense olignonucleotide for the treatment of Angelman syndrome (AS), where she served at the Chief Operating Officer prior to its acquisition by Ultragenyx Pharmaceutical in 2022. After her daughter, Quincy was diagnosed with this rare neurogenetic disorder at the age of five and a half months, Dr. Berent embarked on a herculean effort through FAST to find a treatment. Backed with a medical education and a scientific understanding, while propelled by hope and passion and an incredible scientific team, she felt deeply that a treatment for this condition was possible, despite there being no approved therapeutics. Through the foundations investment in the discovery of numerous therapeutic platforms with academic research groups one program at Texas A&M University discovered a unique genetic region that could be exploited, essentially turning on a silent copy of the gene that is otherwise missing in the neurons of patients with AS. Through her personal determination, GeneTx Biotherapeutics completed the US FDA’s Investigational New Drug (IND) program in incredible time. The first exploratory in vivo studies through to the final in vivo good laboratory practice (GLP) toxicity studies took exactly 13 months, completing an incredibly robust IND package for FIH application. Dr. Berent now consults with and supports various rare disease organizations and companies advancing disease modifying, cell and gene therapies for Angelman syndrome and other neurodevelopmental disorders and is changing the landscape for neurodevelopmental disorders.

 Session Abstract – PMWC 2023 Silicon Valley

Track 1 - January 25 9.00 A.M.-4.15 P.M.

Track Co-Chairs:
Yael Weiss, Mahzi
Peter Marks, FDA

  • Welcome and Opening by Track Chairs: Yael Weiss, Mahzi
  • PMWC 2023 Luminary Award Ceremony
    - Allyson Berent, Angelman Foundation
    - Terry Pirovolakis, CureSPG50
  • From Bench to Bedside - Biomarkers, Natural History Studies, Animal Models (PANEL)
    Chair: Peter Marks, FDA (Allyson Berent, FAST)
    - Emil Kakkis, Ultragenyx
    - Alysson Muotri, UCSD
    - Nasha Fitter, Invitae
  • Commercialization - Moving from Rare to Common (PANEL)
    Chair: Matthew Patterson, Vor Bio
    - Shankar Ramaswamy, Kriya
    - Rahul Singhvi, Resilience
    - Sarah Glass, Ultragenyx
    - Andrew Lo, MIT
    - Mark Harrison, University of British Colombia
  • Future Modalities (PANEL)
    Chair: Shannon Muir, CIAPM
    - Nadav Ahituv, UCSF
    - Amber Salzman, Epic Bio
    - Ben Oakes, Scribe Therapeutics
  • PMWC NCI Showcase
    - Michael M. Binkley, OpenCell
  • Cellular Rejuvenation Programming
    - Hana El-Samad, UCSF
  • Bioethics & Ethical Implications in Cell and Gene Therapies
    - Hank Greely, Stanford
  • Patient Driven Therapies: The Challenges and Successes (PANEL)
    Chair: Yael Weiss, Mahzi
    - Terry Pirovolakis, CureSPG50
    - Allyson Berent, Angelman Foundation
  • PMWC Showcase - Peter Marinkovich, Stanford


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